The EU regulation on orphan medicinal products is intended to encourage the development of drugs that may provide a significant benefit to patients suffering from rare (affecting fewer than five out of 10,000 people) and life-threatening or chronic debilitating conditions for which there is no effective therapies available. The orphan drug designation offers important incentives such as free protocol assistance (to optimize drug development) at the European Medicines Agency (EMA), fee reductions for various regulatory activities and, following drug approval, a grant of 10 years’ market exclusivity in the EU.